CAPR - Earnings call transcript Q2 2022 | Capricor Therapeutics, Inc.

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Operator

Good day and welcome to the Capricor Therapeutics' Second Quarter 2022 Earnings Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Mr. A.J. Bergmann, CFO. sir. ahead, go Please

Anthony Bergmann

good and afternoon. you Thank

regarding, I from Before utilization we potential candidates, uses These milestone of to the and our payments; assumptions, and existing statements uncertainties that that change filings, and investment our data; our are to product current like subject additional candidates, possible resources. statements These be information, based anticipated involve are on we statements our safety, of making intended include forward-looking presentation. a start, will risks and today's our of regarding that certain expectations regulatory and state during cash things, including our number plans, in filings regulatory our would reports. forward-looking conduct and and other among and in may efficacy, differ preclinical actual developments of forward-looking product the to report including These timing periodic future described may present cause SEC, other studies; quarterly clinical or R&D results are the those contained potential risks annual involving and our our with materially and plans made statements. to plans statements

to cautioned that, to we reliance statements undue You disclaim CEO. to statements. Marbán, I'll Linda such on forward-looking any these not are turn place the over obligation update With and call

Linda Marbán

exosome program our Today conference afternoon forward. provide and and Good our thank technology, updates muscular you priorities for as our I outline will call. dystrophy path our for quarter XXXX as us second and joining on well Duchenne platform

Duchenne clinical DMD treating and and is therapeutic across comprises DMD for in young our about and or non-ambulant most stages about very This half course, key boys on and To known to been we options whom two has XX,XXX and largely who population our the XXX proven muscular DMD United be achieved We've successful initiative program, current as CAP-XXXX otherwise the milestones productive over few are disease, you, DMD. quarter lead to-date. later States. at This their dystrophy, the the trials has remind of of CAP-XXXX aimed safe of patients patients completed exist. and in notably, men in treatment of for fronts several many in patient group well-tolerated clinical

Now, updates. of let through me walk some key the and you highlights recent

First, turning XX initiation study United approximately we sites HOPE-X that initiated States. quarter, centers. and III was which study, inform activation in site today, reported and the to our double-blind of goal you very HOPE-X, I randomized selection pleased ongoing Phase July, to investigative with a XX the XX in enrolled seven enroll second to patients patients. as have which In the at of we enrollments certified included placebo-controlled of to am the pivotal Duchenne is

and interested have of growing Annual Conference candidates in our presented recruitment of Project the We now this in PPMD June, a that Phase amplifying in optimistic we Muscular breaking the HOPE-X, published last in open study, with our recent which HOPE-X extension late are we II year, at momentum Dystrophy, the interest year's Lancet trial. label are the The Parent for was will data list or together gain trial.

label third on therapeutic underscore the Our be builds quarter are to reported continue the data, current by sooner. CAP-XXXX HOPE-X recently of it's highlight safety and and of to complete extension efficacy. The projections for the potential open XXXX enrollments promise which of sustained or HOPE-X

be clinical our their its each highlight extension data to relevance and study, a Let clinical was of used me to for allows recap patient to you development the label strategy unique CAP-XXXX. own HOPE-X regulatory that as open which control. very

behavior every but orphan to also function both shown clinical the statistical upper believe that recovered, on fact our and this based upper We upper matter at days. showed originally RMAT, on Then of what rate importance also no limb the CAP-XXXX HOPE-X. notable patients what one of all data gap that All be extension originally conducted where statistical muscle, or this treatment published. that started must function that benefit, of which those group the because limb of and CAP-XXXX. into declined portion non-ambulant results gap the went be all Advanced trial of group the benefit because function group the XX%, function interesting in off cannot and call due of better What label one patients phase, to CAP-XXXX disease was in with open CAP-XXXX earlier this during to FDA, placebo. that of they is based Medicine that in patients DMD, mentioned people limb upper are improvement better limb Those the Then, placebo original declined year. to is but in disease upper in on as placebo progression the up They trial DMD. therapeutic. approximately and year in disease function of dataset, year is did, they we data attenuated CAP-XXXX, received off power same time with potential the designation, its patients and one modifying therefore, regulatory part we entered label went Therapy Regenerative also the were with the exemplary off a patients with and with significant a This they received group therapy Hope-X and and group open designations lose were one and of limb most is finished had on First, clinically better was function.

this our requesting We approval. we year further potential label with forward this are towards occur should a FDA, regulatory believe meeting data, extension will which support which path to open present

remind complement and Upper X.X extension. trial. BLA label This HOPE-X's to validated which planning are endpoint results However, muscle remain which in significant meeting function this tool performance the HOPE-X skeletal meeting, pivotal measure trial, HOPE-X submission, in to non-ambulant clinical assess is executing on primary be we to we To we you focused to a in slated patients, hold Limb the is open well. is our our also on which saw year a prior will and later which the CMC required and as statistically HOPE-X of

potential Another our preparing of CAP-XXXX for priority key up program future for the involves the manufacturing. launch, scale including commercial

see supplying site current to Diego and by our support versatile Angeles our facility a as focused converting labs manufacturing are clinical a our While We is cost-effective early commercial our HOPE-X manufacturing San in supplementing fully potential we trial, measure. portion of on Los efforts this launch.

encompasses for we plan at important part manufacturing our scaled have future Additionally, commercial plan an may Lonza be the up CAP-XXXX. our work done as they of

NS the our community eligible know, in and well-resourced trusted support you a CAP-XXXX leadership from caregivers. respected States. believe with the entered Nippon distribution in United experienced benefit commercial As we its has who to enriching well, DMD and this has approved, for already U.S. a Shinyaku's commercial an been and more Shinyaku infrastructure we patients could agreement If Pharma, a therapy. space patients established their will serve partner Nippon partner subsidiary and into Pharma and NS

will milestone a potential benefit the To if to upfront has up and CAP-XXXX we reminder, some opportunities. be and a $XX to achieved, paid Capricor's payment HOPE-X. Nippon course ex-U.S. Capricor came payments, reach million million explore a of reach of patients continue to As of Shinyaku which, the maximize agreement in during to with with $XXX globally, partnership potential will

bring business CMC, execute to continue possible. our to goal as and CAP-XXXX in as above, outlined just clinical, committed patients on Our to quickly development are regulatory, to as I is goals as we

now and a also platform exosomes focused on on been drug delivery technology. Turning has to year identifying The potential our exosomes for developing as versatile last applications.

significant with having to manufacturing lipid a in to made exosomes of progress potential have cell targeted of be competitive as a the type. We specific to systems or additional other delivery the biomarker the alternative benefit

via preservation drive relies RNAs, platform allows We believe loading targeted to modalities. have the this applications potential attract similar and therapeutic and exogenous multiple Our other cargo. therapeutic of endogenous platform what payloads. proteins. vaccines, molecules, consistency and for as delivery payloads, positions that types of for be are in technology, to which approach new on are able potential This as of our of including partnerships doing, or small loading process our domains, better Capricor the certain interfering delivery integrity an will including The payloads which for carry well of other in payloads, emerging via proprietary exosomes process produced RNAs, antisense most including last is space to can types an

have programs, potential the update to for future turn now vaccine of efficiently prioritizing carries We CFO I is exosomes on the our for over to we milestones. have and with our our which to the to At cash important positioning related current platform we the call deliver A.J. detailed XXXX present, quarter focus us on our continue our financials. preclinical will By second an develop while proprietary robust core on for plan of CAP-XXXX We to data. into position, Bergman, more for utilize on ability opportunity political exosome-based our used the technology discussion. partnering this technology DMD, opportunities. develop pipeline commercialization

Anthony Bergmann

you, our provided on summary financials Thank XXXX a Linda. of GAAP press of release quarter second afternoon's This a basis.

on to quarterly which shortly report become also the expect will XX-Q, and accessible as section You refer the website. SEC our the company website, to of may on as well Form financial be available we

December company's and marketable compared to on million approximately XXth, $XX.X of XXst, approximately cash cash, the XXXX, $XX.X XXXX. As million equivalents, totaled June securities

second and our equivalents, will As available anticipated expect that the plan and of capital on current into and Linda quarter financial we XXXX. sufficient believe securities mentioned, marketable based to expenses cover requirements that resources, cash, be operating cash our we

Turning by $XX now upfront XXXX, the payment approximately by of activities net to provided was operating half Shinyaku. in the our $XX.X cash from million, first million Nippon driven the financials

quarter our development compensation compared million in For second QX and research approximately of $X.X expense million stock-based approximately to $X.X XXXX, expenses, XXXX. was excluding the

was Net million general now approximately in will of XXXX million that, compared the XXXX Excluding approximately $X.X half line $X.X for approximately of the and to we loss and a net loss open the XXXX. for XXXX. million in With approximately expense compensation, our stock-based first million half QX $XX.X $X.X of was administrative first questions. up QX for

Operator

Pantginis Joe the ahead, of your take with Thank from line Instructions] now H.C. our question you. first open. is go We'll Please Wainwright. line [Operator

Joe Pantginis

A.J. afternoon. and Linda question. Thanks Good the for taking Hi

actually. a So, couple questions

get more color first, or little a So, detail. wanted to

data? present what primary looking Are come have goal it to dynamic, launch FDA the is potential describe to you is out you be the the I will to said the the ultimate the hope FDA change what but goal here? mean, potentially obviously, earlier? OLE predicated you to market would answers or you goal how where of get meeting, Now, wish-list on what for says, upcoming your your

Linda Marbán

our you Yes, I Joe I'm you. ultimate to looking talking in to and of -- goal, September. mean, great first all, seeing it's forward

DMD to as to possible. is and goal markets market get ultimate Our to CAP-XXXX quickly these as

by soon As extension as the of open time decline -- of went go in course the is steady as now data think what they to then as I delay of this a is them We're We they we said XX%. back The with for as the and patients I've work to as for possibly off been about talk it get delay saying the history extraordinary. my in CAP-XXXX, FDA is the CAP-XXXX, on they activity. became think as is placebo decline on can. a muscle and the gap quickly we soon to better. straight on CAP-XXXX, year few we clear bring on data process show weeks' very of they were disease and saw representative label natural to data excited the when decline are modifying this strong and years, and they approval going placebo the similar to to two on disease We this year were

Joe Pantginis

speak certainly thinking it another about data so I'm in itself in I'll guess, muscle it. the for as I ask way, And my belief,

you'd call just dynamic, fences does regard I prior to to a CAP-XXXX program regard mean, market market, the to the for on the swing maybe the it data of potential I like mean, HOPE-X? potential the access with is to commercialization readout And basis the a managed based there So, be at on I with this to the point let's when so a something have patient-by-patient on look present? say on

Linda Marbán

and thing very feedback, of the the we'll BLA-ready, are CAP-XXXX presenting the we now preferred blocks Yes, access our so label best regulatory building ready get getting goals FDA, in our sure commercialization. got are right exciting to RMAT, we there to -- focused, open decide for then data FDA the with to means our the their That are CMC, meeting the regarding making place extension really designation forward. forward what's path that path The FDA. with

So, the they're going definitely And And anecdotally, the this. data feeling certainly everybody excited are is with that functioning to, work we've patients and on to better. us they're saying it. by shown

And so shout going same to -- stand say the they're and and even sometimes up thing.

So, got the knows, not to place. team in fences anybody fan, a I manager swinging We've And certainly, the the know do. so we've baseball put I'm afraid is for there. big something I'm if

Joe Pantginis

you go. There

you out regard planning a I of needs those Okay. timing potentially project concomitant regard you ahead standpoint? meeting with then, guess, lab, made from would glad regard your I'm or separate San glad CMC, of you're, either with to And How a manufacturing supplementing meeting manufacturing, you're with to that your the beyond Diego and comments. to I'm

Linda Marbán

the terms on plan of working that In we're now. manufacturing, right

And product facility, We'll our have option, this not NS, partner San only commercial of we're as apparent. for We for move our affords Diego getting ready evaluate opportunity low us announced with forward. our tremendous we'll which as relatively as for the it just that becomes a the building that need on along BLA, here, cost a I we updated also is as for part plans it keep launch. everybody but more labs

Joe Pantginis

discussed. of obviously, And has because it. future exosomes then Got being a lastly, just the lot

see COVID said you've candidate tipping you're previously? candidate, hand. partner that be And for you to a looking your So, get that in we and things data, what you form a about in of your without candidate that in from too, your visibility things here release necessarily you more some little also vaccine preclinical have kind something going the is potential discussed mentioned maybe of press a couple apart might

Linda Marbán

of sort it's the COVID on Yes, twist a candidate.

to level. it's we're too can about really with teams But so right a in system been it this our every you So, the standardized delivery sure because now will protected make I on much up the able What be inside. a protein has the The recombinant a be long I legal and IP them can vaccine, with we've but a hard we targeting competitive lipid it they put going manufacturing of payloads current vaccine can very into way put on which can we're exosomes, -- can't scaled takes is of we being for quickly. candidate outside, you very and working talk such, molecule make and very candidates exciting that made to about know manufactured really any on is out to types the vaccine advantage similar to there. be time, on custom that because working we've so tell the of other

on infectious in where might we've reality, types could other or for and So, translated working been any of, we system, disease while, that rapidly necessary utility, of a or to vaccine our use be available a desired. protein-based COVID, model sort as be something but

Joe Pantginis

kind And it's last mind it. I right it's Got the looks environment, just king important, starting question, don't is my biotech back, and to cash of based bounce you but if now. on which think current actually like

speaks to able have being quarter a second of the XXXX balance volumes. into So,

we be the that you what exosome program, of one consider? about further that to address or externally press of in at your external potential would look of one I source A.J.'s things to this -- guess uses the kind of wanted expand or capital, So, release excluding expand could pipeline I from your and is would comments comments this strategic if options something look an an

Anthony Bergmann

obviously, bit cash Yes, take our Joe, of little spend. that judicious question. I happy to a mean, being we're with

DMD, exposome quarter, status. I a of because have a just really CAP-XXXX XXXX. we're is nice what on we think in and now runway mission our for prioritizing preclinical what second the the into right articulated focused we pipeline And current

our there, all forward. we're it's out effectively obviously going our rate but moving we're manage considering burn So, best to options, and to do

Joe Pantginis

guys. you Thank it. Got

Linda Marbán

Take Joe. care. Thanks

Anthony Bergmann

question? we do Operator, another have

Unidentified Analyst

Hello.

Anthony Bergmann

Hi, Alan.

Operator

[Operator Instructions]

Unidentified Analyst

Hello.

Operator

Yes. Alan. Go It's ahead open.

Unidentified Analyst

I'm something like a sorry, to its continues period. that, was interim off-treatment for How it hear descent for an might Linda, need period, the you. do if didn't to provide it And good with for look And A.J., talk to that And there treatment for the about free feel even pursue anything couldn't I'd CAP-XXXX the Okay, you restored. interim is was treatment continuous like the go you mentioned after had and additional the off-time envision treatment? bit. thoughts to that condition I any earlier. Linda functioning like looks during group

Linda Marbán

on you. HOPE-X, Yes, we're forward. clinical thanks, what looking that's will know emphasize called talk that we get What answering and they the don't to lost, delays the Alan, to be disease. me the appears we we time let muscle, believing program it's progression once forward function that we're of is Obviously, see first again, great lost. continue HOPE-Duchenne this the to But is to move HOPE-X to a CAP-XXXX as question being from program back and it

delay delay progression, we about XX%. we So, it

temporal months. over that, the decline. you function, you preserving that way, out we're year time, can of If of trajectory in And think of in it's eight you to that very when up sort a a of about think significant want a add improvements

been are We skipper, therapies CAP-XXXX discussed, the gene and therapeutics pathway of DMD. the on perform exon believe with that coming and along based development with the mechanism published its for which has also action, will potential other

going but So, both or a be it's one to other, scenario. a not the

can, we your approximately XX for life therapeutic, now that visioning away patients fact of typically that's right if to is they XX kind a XX dosing non-ambulant, the just which between make of seeing age years to everything that forward well. highlight looking XX,XXX years which one the way a four kind are hopefully. and is longer DMD This XX,XXX and half pass when XX right for much until of of, now patients year eligible or of the patients with years boys, be very started of now times could we're age, steady rest going works so maintain And

therapeutic safe So, this very is for a DMD, option as well. long-term

Unidentified Analyst

a you If or CAP-XXXX. as -- backup you a the time lines term, do term on some for the United just commercial Lonza? A on as something in fine ask as adding even expand I you longer to at can Or intent elsewhere? see the it see capacity same as longer co-manufacturing you and continue States larger

Linda Marbán

label on As open It Alan, in fast-forward. incredibly terms our we were imagine, you literally put preparing by launch the extension for can excited commercial data. program of

We are up for going sort be enacted. of scaling manufacturing. the and already place, options They've in now how scaling out evaluating building that's put been blocks to

into market, closer to be it that labs move in get decided to will our now are San commercial make we is to reason doses to us we this to allows that. Diego As build better here facility begin the assess because right we The as we able that ready.

So, with we're to getting as for regulatory CAP-XXXX patients that's as And as hopefully us rapidly strategy, already as our preparing into that launch. needed going commercial possible. well help

Unidentified Analyst

Rooting trial. again. like about this slide and around patients talked I'd from to they're actually tracking you. are you world excited to I and I the for

and soon. to see luck you good So, hope

Linda Marbán

well. Be Alan. Thanks

Anthony Bergmann

have Operator, difficulties. might Alan. technical some Thanks you think I

that's -- you If to the end closing the questions, some think of remarks. we're I going going we're turn and the can to make us, hear

Linda Marbán

in the about company where the the Cell Vesicle-Based October, updates both including program. interactions months, well we FDA we joining we updates Therapeutic with CAP-XXXX the progress Conference, further on presentations. in the Extracellular provide are Medicines you as as well our Summit several In We're more than today. provide plan in Development we our Fitzgerald on in at as September, pleased and being will H.C. Global encouraged Care over Genetics engineered to Wainwright ever Thank of our Health to and summary, next continue our outlook for several are as various exosomes the with conferences on QX progress us held featured Cantor Conference, and

we way, for Before investors, our along team I conclude thank have patients the Capricor, Once you and like to call, people our who us joining been including would again, thank supportive families. our the today. today's and their at many

Operator

you conference. concludes Thank your This today's for participation.

You disconnect. may now

Capricor Therapeutics (CAPR) 11 Aug 222022 Q2 Earnings call transcript